Evolving assessment pathways for precision oncology medicines to improve patient access: a tumor-agnostic lens.

BACKGROUND: Genomic and molecular alterations are increasingly important in cancer diagnosis, and scientific advances are opening new treatment avenues. Precision oncology (PO) uses a patient's genomic profile to determine optimal treatment, promising fewer side effects and higher success rates. Within PO, tumor-agnostic (TA) therapies target genomic alterations irrespective of tumor location. However, traditional value frameworks and approval pathways pose challenges which may limit patient access to PO therapies. OBJECTIVES: This study describes challenges in assessing PO and TA medicines, explores possible solutions, and provides actionable recommendations to facilitate an iterative life-cycle assessment of these medicines. METHODS: After reviewing the published literature, we obtained insights from key stakeholders and European experts across a range of disciplines, through individual interviews and an industry workshop. The research was guided and refined by an international expert committee through 2 sounding board meetings. RESULTS: The current challenges faced by PO and TA medicines are multiple and can be demonstrated through real-world examples of the current barriers and opportunities. A life-cycle approach to assessment should be taken, including key actions at the early stages of evidence generation, regulatory and reimbursement stage, as well as payment and adoption solutions that make use of the evolving evidence base. Working toward these solutions to maximize PO medicine value is a shared responsibility and stands to benefit all stakeholders. CONCLUSIONS: Our call to action is to expand access to comprehensive genomic testing, foster a learning health care system, enable fast and equitable access to cost-effective treatments, and ultimately improve health outcomes.

A Logic Model Framework for Planning an International Refugee Health Research, Evaluation, and Ethics Committee.

Collaborative approaches to supporting the health of refugees and other newcomer populations in their resettlement country are needed to address the complex medical and social challenges they may experience after arrival. Refugee health professionals within the Society of Refugee Healthcare Providers (SRHP)-the largest medical society dedicated to refugee health in North America-have expressed interest in greater research collaborations across SRHP membership and a need for guidance in conducting ethical research on refugee health. This article describes a logic model framework for planning the SRHP Research, Evaluation, and Ethics Committee. A logic model was developed to outline the priorities, inputs, outputs, outcomes, assumptions, external factors, and evaluation plan for the committee. The short-term outcomes include (1) establish professional standards in refugee health research, (2) support evaluation of existing refugee health structures and programs, and (3) establish and disseminate an ethical framework for refugee health research. The SRHP Research, Evaluation, and Ethics Committee found the logic model to be an effective planning tool. The model presented here could support the planning of other research committees aimed at helping to achieve health equity for resettled refugee populations.

Cost-Effectiveness Analysis of Sequential Biologic Therapy with Ixekizumab Versus Secukinumab in the Treatment of Active Psoriatic Arthritis with Concomitant Moderate-to-Severe Psoriasis in the UK.

BACKGROUND: Interleukin-17A (IL-17A) antagonists are a recent innovation for treating psoriatic arthritis (PsA). There are currently no cost-effectiveness analyses (CEAs) comparing the IL-17A antagonists ixekizumab and secukinumab in PsA from a UK perspective. OBJECTIVE: We conducted a CEA from the UK National Health Service perspective to compare ixekizumab versus secukinumab in patients with PsA and concomitant moderate-to-severe plaque psoriasis. METHODS: A Markov model was developed based on the widely accepted York model. In biologic disease-modifying antirheumatic drug (bDMARD)-naïve patients, ixekizumab → ustekinumab → best supportive care (BSC) was compared with secukinumab → ustekinumab → BSC. For bDMARD-experienced patients, ixekizumab → BSC was compared with secukinumab → BSC. At the end of the bDMARD trial period, Psoriatic Arthritis Response Criteria (PsARC) responders continued to receive the bDMARD in the continuous treatment period. PsARC nonresponders and patients who ceased continuous treatment transitioned to the trial period of the next treatment. RESULTS: Ixekizumab was less costly and provided more quality-adjusted life-years (QALYs) than secukinumab in bDMARD-naïve and -experienced patients based on list prices, although cost savings and QALY gains were small to modest. In bDMARD-naïve patients, total costs were £155,455 compared with £155,530 for secukinumab (year 2017 values). Total QALYs were 8.127 versus 7.989. In bDMARD-experienced patients, the corresponding values were £140,051 versus £140,264 for total costs and 3.996 versus 3.875 for total QALYs. CONCLUSION: Ixekizumab provided more QALYs at a marginally lower cost than secukinumab, and the results were most sensitive to changes in drug costs. Other factors, such as patient preferences for the number of injections and confidential price discounts, may be important considerations in clinical decision-making.

Qualitative research study on addressing barriers to healthy diet among low-income individuals at an urban, safety-net hospital.

BACKGROUND: Some American households experience food insecurity, where access to adequate food is limited by lack of money and other resources. As such, we implemented a free 6-month Fruit and Vegetable Prescription Program within a large urban safety-net hospital. METHODS: 32 participants completed a baseline and postintervention qualitative evaluation about food-related behaviour 6 months after study completion. Deductive codes were developed based on the key topics addressed in the interviews; inductive codes were identified from analytically reading the transcripts. Transcripts were coded in MAXQDA V.12 (Release 12.3.2). RESULTS: The information collected in the qualitative interviews highlights the many factors that affect dietary habits, including the environmental and individual influences that play a role in food choices people make. Participants expressed very positive sentiments overall about their programme participation. CONCLUSIONS: A multifaceted intervention that targets individual behaviour change, enhances nutritional knowledge and skills, and reduces socioeconomic barriers to accessing fresh produce may enhance participant knowledge and self-efficacy around healthy eating. However, socioeconomic factors remain as continual barriers to sustaining healthy eating over the long term. Ongoing efforts that address social determinants of health may be necessary to promote sustainability of behaviour change.

Cost-effectiveness analysis of sequential biologic therapy with ixekizumab versus secukinumab as first-line treatment of moderate-to-severe psoriasis in the UK.

AIMS: Patients with psoriasis often undergo treatment with a sequence of biologic agents because of poor/loss of response to initial therapy. With the availability of newer agents like ixekizumab and secukinumab, there is a need for cost-effectiveness analyses to better reflect current clinical practice. This study aimed to assess the cost-effectiveness of a sequence of biologic therapies containing first-line ixekizumab vs first-line secukinumab in patients with moderate-to-severe plaque psoriasis in the UK. MATERIALS AND METHODS: A Markov model with a lifetime horizon was developed to compare the cost-effectiveness of ixekizumab and secukinumab treatment sequences: ixekizumab → ustekinumab → infliximab → best supportive care (BSC) vs secukinumab → ustekinumab → infliximab → BSC. The model used monthly cycles, and included four health states: trial period, treatment maintenance, BSC, and death. At the end of the trial period, responders transitioned to maintenance therapy; non-responders transitioned to the next biologic in the sequence. An annual discontinuation rate of 20% was assumed for maintenance therapy. RESULTS: The ixekizumab sequence provided cost savings of £898 (£176,203 vs 177,101) [year 2015 values] and gained 0.03 more quality-adjusted life-years (QALYs: 1.45 vs 1.42) vs the secukinumab sequence over the lifetime horizon. Probabilistic sensitivity analysis showed an 89.8% likelihood that the ixekizumab sequence would be cost-effective at a threshold of £20,000 per QALY gained. LIMITATIONS: The analysis used list prices for drugs rather than confidential, preferentially priced Patient Access Scheme costs. In addition, efficacy input data were based on a network meta-analysis, as there were no head-to-head trials comparing ixekizumab and secukinumab. CONCLUSION: First-line treatment with ixekizumab as part of a specific sequential biologic therapy for moderate-to-severe plaque psoriasis in the UK provided slight advantages in cost savings and QALYs gained over a similar treatment sequence initiated with secukinumab. In view of the small magnitude of these differences, factors such as patient preferences (e.g. for number of injections) and long-term safety (e.g. related to time on the market) may also be important for clinical decision-making.

Cost-effectiveness of TYRX absorbable antibacterial envelope for prevention of cardiovascular implantable electronic device infection.

AIMS: Infection is a major complication of cardiovascular implantable electronic device (CIED) therapy that usually requires device extraction and is associated with increased morbidity and mortality. The TYRX Antibacterial Envelope is a polypropylene mesh that stabilizes the CIED and elutes minocycline and rifampin to reduce the risk of post-operative infection. METHODS: A decision tree was developed to assess the cost-effectiveness of TYRX vs standard of care (SOC) following implantation of four CIED device types. The model was parameterized for a UK National Health Service perspective. Probabilities were derived from the literature. Resource use included drug acquisition and administration, hospitalization, adverse events, device extraction, and replacement. Incremental cost-effectiveness ratios (ICERs) were calculated from costs and quality-adjusted life-years (QALYs). RESULTS: Over a 12-month time horizon, TYRX was less costly and more effective than SOC when utilized in patients with an ICD or CRT-D. TYRX was associated with ICERs of £46,548 and £21,768 per QALY gained in patients with an IPG or CRT-P, respectively. TYRX was cost-effective at a £30,000 threshold at baseline probabilities of infection exceeding 1.65% (CRT-D), 1.95% (CRT-P), 1.87% (IPG), and 1.38% (ICD). LIMITATIONS AND CONCLUSIONS: Device-specific infection rates for high-risk patients were not available in the literature and not used in this analysis, potentially under-estimating the impact of TYRX in certain devices. Nevertheless, TYRX is associated with a reduction in post-operative infection risk relative to SOC, resulting in reduced healthcare resource utilization at an initial cost. The ICERs are below the accepted willingness-to-pay thresholds used by UK decision-makers. TYRX, therefore, represents a cost-effective prevention option for CIED patients at high-risk of post-operative infection.

Long-Term Cost-Effectiveness of Transanal Irrigation in Patients with Neurogenic Bowel Dysfunction.

BACKGROUND: People suffering from neurogenic bowel dysfunction (NBD) and an ineffective bowel regimen often suffer from fecal incontinence (FI) and related symptoms, which have a huge impact on their quality of life. In these situations, transanal irrigation (TAI) has been shown to reduce these symptoms and improve quality of life. AIM: To investigate the long-term cost-effectiveness of initiating TAI in patients with NBD who have failed standard bowel care (SBC). METHODS: A deterministic Markov decision model was developed to project the lifetime health economic outcomes, including quality-adjusted life years (QALYs), episodes of FI, urinary tract infections (UTIs), and stoma surgery when initiating TAI relative to continuing SBC. A data set consisting of 227 patients with NBD due to spinal cord injury (SCI), multiple sclerosis, spina bifida and cauda equina syndrome was used in the analysis. In the model a 30-year old individual with SCI was used as a base-case. A probabilistic sensitivity analysis was applied to evaluate the robustness of the model. RESULTS: The model predicts that a 30-year old SCI patient with a life expectancy of 37 years initiating TAI will experience a 36% reduction in FI episodes, a 29% reduction in UTIs, a 35% reduction in likelihood of stoma surgery and a 0.4 improvement in QALYs, compared with patients continuing SBC. A lifetime cost-saving of £21,768 per patient was estimated for TAI versus continuing SBC alone. CONCLUSION: TAI is a cost-saving treatment strategy reducing risk of stoma surgery, UTIs, episodes of FI and improving QALYs for NBD patients who have failed SBC.

Healthful Nutrition of Foods in Navajo Nation Stores: Availability and Pricing.

PURPOSE: Low availability and affordability of healthier foods in food stores on the Navajo Nation (NN) may be a community-level risk factor for the high prevalence of obesity among the Navajo people. This study assessed the availability and pricing of foods and beverages in supermarkets and convenience stores throughout the NN. DESIGN: Descriptive study design using the Nutrition Environment Measurement Survey in Stores audit tool. SETTING: Supermarkets (n = 13) and convenience stores (n = 50) on NN and border-town supermarkets (n = 9). SUBJECTS: Not applicable. MEASURES: Availability and pricing of healthy and less-healthy foods. ANALYSIS: Descriptive and χ(2) analyses. RESULTS: Navajo convenience stores offered fewer healthier food options compared to Navajo supermarkets. In Navajo convenience stores, 100% whole grain products, reduced-fat cheese, lean meats, reduced-fat chips, and fat-free or light hot dogs were available in fewer stores than their corresponding less-healthy versions (all with p < .05). In both Navajo supermarkets and convenience stores, 100% whole wheat bread, lean cold cuts, and reduced-fat cheese were all more expensive per unit than their corresponding less-healthy versions (all with p < .05). CONCLUSIONS: According to this study, healthier foods are not as readily available in Navajo convenience stores as they are in Navajo supermarkets. Improving access to and affordability of healthier foods in reservation stores of all sizes may support healthy eating among Navajo residents.

Cost-effectiveness of first-line induction and maintenance treatment sequences in non-squamous non-small cell lung cancer (NSCLC) in the U.S.

OBJECTIVES: Due to the lack of direct head-to-head trials, there are limited data regarding the comparative effectiveness of induction-maintenance sequences. The objective of this study was to develop a cost-effectiveness model to compare induction-maintenance sequences in the US for the treatment of advanced non-squamous NSCLC. MATERIALS AND METHODS: Decision analytic modelling was used to synthesize the treatment effect and baseline risk estimates for nine induction and maintenance treatment sequences, reflecting treatments used in the US. The model was structured using an area-under-the-curve approach and sensitivity analyses were conducted. Model validation was conducted by an independent third party. RESULTS: All active maintenance therapy-containing regimens, with the exception of gemcitabine+cisplatin (first-line)→erlotinib (maintenance), were more costly than induction-only regimens. Concerning treatments that may be cost effective, the incremental costs per life-year gained were $121,425, $148,994, and $191,270 for gemcitabine+cisplatin→erlotinib versus gemcitabine+cisplatin→best supportive care (BSC), pemetrexed+cisplatin→BSC versus gemcitabine+cisplatin→erlotinib, and for pemetrexed+cisplatin→pemetrexed versus pemetrexed+cisplatin→BSC, respectively. All other regimens were found to be dominated (carboplatin+paclitaxel→BSC; carboplatin+paclitaxel→erlotinib; carboplatin+paclitaxel→pemetrexed; bevacizumab+carboplatin+paclitaxel→bevacizumab) or extendedly dominated (cisplatin+gemcitabine→pemetrexed). Sensitivity analyses demonstrated stability. CONCLUSIONS: Depending on the specific cost-effectiveness threshold used by a decision maker, the most cost-effective treatment sequence may include the referent comparator gemcitabine+cisplatin and the studied regimens of gemcitabine+cisplatin→erlotinib, pemetrexed+cisplatin→BSC, or pemetrexed+cisplatin→pemetrexed.

Healthful food availability in stores and restaurants--American Samoa, 2014.

American Samoa, one of the U.S.-affiliated Pacific Islands, has documented the highest prevalence of adults with obesity (75%) in the world. The nutritionally poor food and beverage environment of food retail venues has been suspected to be a contributing factor, although an evaluation of these venues in American Samoa has not been conducted. In January 2014, American Samoa established an Obesity Task Force to develop policies and strategies to combat obesity. To inform the efforts of the task force, the American Samoa Department of Health and CDC conducted a baseline assessment of the availability, pricing, and promotion of healthful foods at retail food venues. Previously validated food environment assessment tools were modified to incorporate American Samoa foods and administered in a geographically representative sample of 70 stores (nine grocery stores and 61 convenience stores) and 20 restaurants. In convenience stores, healthful items were not found as available as less healthful counterparts, and some healthful items were more expensive than their less healthful counterparts. For restaurants, 70% offered at least one healthful entrée, whereas only 30% had healthful side dishes, such as vegetables. Actions to promote healthy eating, such as providing calorie information, were rare among restaurants. Improving availability, affordability, and the promotion of healthful foods in American Samoa stores and restaurants could support healthy eating among American Samoa residents.

Self-reported advertising exposure to sugar-sweetened beverages among US youth.

OBJECTIVE: According to the Federal Trade Commission, in 2009, the top food category with teen-directed marketing expenditures was sugar-sweetened beverages (SSB). The present study reports on exposure to SSB advertisements using self-report data from adolescents. DESIGN: Cross-sectional study design using descriptive statistics to assess self-reported frequency of exposure to SSB advertisements and multivariable logistic regression to examine associations between frequency of SSB advertising exposure and sociodemographic variables. SETTING: Online survey conducted at home. SUBJECTS: US adolescents aged 12-17 years (n 847). RESULTS: Among the surveyed adolescents, 42 % to 54 % reported seeing/hearing SSB advertisements ≥1 time/d. Those aged 14-15 years were more likely to report seeing/hearing soda, sports drink and energy drink advertisements ≥1 time/d than 16- to 17-year-olds. Males were more likely to report seeing/hearing sports drink advertising ≥1 time/d than females. Non-Hispanic black adolescents were more likely to report seeing/hearing fruit drink and sports drink advertisements ≥1 time/d than non-Hispanic white adolescents. Adolescents whose parents had high-school education or less were more likely to report seeing/hearing soda, fruit drink and energy drink advertisements ≥1 time/d than adolescents whose parents were college graduates. CONCLUSIONS: Almost half of the adolescents sampled reported daily SSB advertising exposure, with higher exposure among African Americans and adolescents with less educated parents. These data can help inform potential actions that decision makers might take, such as education of adolescents and their caregivers on the potential impact of beverage advertising, especially among groups at higher risk for obesity.

Association between reported screening and counseling about energy drinks and energy drink intake among U.S. adolescents.

OBJECTIVE: Possible adverse health consequences of excessive energy drink (ED) consumption have led to recommendations by the American Academy of Pediatrics discouraging ED intake by youth. However, limited information on ED counseling by health care providers exists. METHODS: Data was obtained from the 2011 YouthStyles Survey administered to youth aged 12-17 (n=815). The outcome variable was ED consumption (none vs. ≥1 time/week) and exposure variables were screening and counseling about ED (if doctor/nurse asked about ED consumption and if doctor/nurse recommended against ED consumption). RESULTS: Approximately 8.5% of youth consumed energy drinks weekly, 11.5% reported being asked by their doctor/nurse about frequency of ED consumption, and 11.1% were advised by their doctor/nurse against ED intake. Multivariable logistic regression analysis revealed that the odds for drinking ED ≥1 time/week was significantly higher in youth who were asked how often they drank ED by their doctor/nurse (odds ratio=2.46) vs. those who were not asked. CONCLUSION: About 1 in 9 youth reported receiving counseling discouraging ED consumption from their doctor/nurse, and a greater proportion of youth who were screened about ED also reported ED consumption. PRACTICE IMPLICATIONS: Efforts by health care providers to educate youth about potential harms of consuming ED are needed.

Effectiveness of case management strategies in reducing emergency department visits in frequent user patient populations: a systematic review.

BACKGROUND: Case management (CM) is a commonly cited intervention aimed at reducing Emergency Department (ED) utilization by "frequent users," a group of patients that utilize the ED at disproportionately high rates. Studies have investigated the impact of CM on a variety of outcomes in this patient population. OBJECTIVES: We sought to examine the evidence of the effectiveness of the CM model in the frequent ED user patient population. We reviewed the available literature focusing on the impact of CM interventions on ED utilization, cost, disposition, and psychosocial variables in frequent ED users. DISCUSSION: Although there was heterogeneity across the 12 studies investigating the impact of CM interventions on frequent users of the ED, the majority of available evidence shows a benefit to CM interventions. Reductions in ED visitation and ED costs are supported with the strongest evidence. CONCLUSION: CM interventions can improve both clinical and social outcomes among frequent ED users.